An Observational Study of SonoVue®/Lumason®-Enhanced Urosonography in Paediatric Subjects with Known or Suspected Vesicoureteral Reflux (VUS for evaluation of Paediatric VUR)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Post-authorization observational, retrospective, comparative study

Study drug and medical condition

• Name of medicine:
  SONOVUE
• Medical condition to be studied:
  Vesicoureteric reflux

Population studied

• Short description of the study population:
  The study will be conducted in subjects below 18 years of age who had undergone contrast enhanced VUS with SonoVue/Lumason or VCUG as part of their standard of care at least 12 months prior to enrollment and have a documented follow-up during the 12 months after the baseline exam.
• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  400

Study design details

• Main study objective:
  To assess subject management decision and changes during a follow-up period of at least 12-months among children undergoing SonoVue/Lumason-enhanced VUS (VUS group) in comparison with children undergoing VCUG (VCUG group) for assessment of VUR.
• Outcomes:
  To describe the severity (grading) of VUR and the type of treatment (conservative or surgery) among subjects in the VUS and the VCUG groups with positive findings. To determine the incidence of recurrent UTIs or breakthrough UTIs during the follow-up period among subjects in the VUS and VCUG groups. To estimate the proportion of technically inadequate imaging procedures for both VUS and VCUG
• Data analysis plan:
  In general, summary statistics (mean, median, standard deviation, minimum, and maximum) will be provided for continuous variables, and the number and percentage of each category will be provided for categorical data. Unless otherwise specified, the analysis will be provided by study group, i.e. VUS and VCUG, and the statistical tests will be 2-sided at 0.05 level of significance. No interim analysis is planned. Missing data will not be imputed in general. Any changes in the original statistical methodology will be documented in the statistical analysis plan. All statistical analyses will be performed using SAS® software.

Documents

Study results

BR1-145 CSR Synopsis